News

In a groundbreaking study, scientists reported editing and correcting, for the first time, a defective gene in human embryos. Additionally, this gene-editing technique appeared to be safely performed without introducing additional harmful genetic alterations. These results represent a key achievement in human genetic engineering and highlight a potential way…

Treatment with Alcresta Therapeutics’ product Relizorb effectively increased fat absorption in patients with cystic fibrosis (CF) receiving enteral tube feeding who have trouble breaking down and absorbing these molecules. This resulted in a reduction of the frequency and severity of gastrointestinal symptoms. These findings were published recently in the…

The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) to treat more than 600 of the estimated 30,000 Americans with cystic fibrosis (CF), Vertex Pharmaceuticals announced. The oral medicine may now be prescribed to CF patients ages 2 and older who have one of five residual…

A U.S. government-backed initiative against drug-resistant bacteria has awarded Antabio $2.8 million to continue developing treatments for Pseudomonas, the most common infection in cystic fibrosis (CF). The grant came from a public-private partnership known as CARB-X, which stands for Combating Antibiotic Resistant Bacteria Biopharmaceutical Accelerator. CARB-X, formed in July 2016,…

Vertex Pharmaceuticals is continuing to develop treatments for all people with cystic fibrosis (CF), with clinical trials exploring combinations of its potentiator and corrector therapies. At the same time, efforts to expand the use of already-approved treatments are ongoing. In addition, the company recently completed the purchase of Concert’s…